Narcolepsy

HARMONY BIOSCIENCES RECEIVES U.S. FOOD AND DRUG ADMINISTRATION ORPHAN DRUG DESIGNATION FOR PITOLISANT IN PRADER-WILLI SYNDROME

Retrieved on: 
Tuesday, February 20, 2024

PLYMOUTH MEETING, Pa., Feb. 20, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of Prader-Willi syndrome (PWS).

Key Points: 
  • PLYMOUTH MEETING, Pa., Feb. 20, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of Prader-Willi syndrome (PWS).
  • "The decision to grant Orphan Drug designation to pitolisant indicates that it could be a promising treatment option for people living with Prader-Willi syndrome," said Kumar Budur, M.D., M.S., Chief Medical Officer at Harmony Biosciences.
  • FDA Orphan Drug designation incentivizes the advancement of promising therapies for rare diseases by providing tax credits for clinical development, waivers for user fees, and seven years of market exclusivity following drug approval.
  • In the upcoming Phase 3 registrational TEMPO study, Harmony will assess the safety and efficacy of pitolisant in treating EDS and behavioral disturbances in PWS.

Takeda Intends to Rapidly Initiate the First Global Phase 3 Trials of TAK-861, an Oral Orexin Agonist, in Narcolepsy Type 1 in First Half of Fiscal Year 2024

Retrieved on: 
Thursday, February 8, 2024

It is currently classified into two different types: narcolepsy type 1 (NT1) caused by significant loss of orexin neurons with resulting lack of orexin, and narcolepsy type 2 (NT2) where orexin levels are generally normal.

Key Points: 
  • It is currently classified into two different types: narcolepsy type 1 (NT1) caused by significant loss of orexin neurons with resulting lack of orexin, and narcolepsy type 2 (NT2) where orexin levels are generally normal.
  • Stimulating the orexin receptor 2 in NT1 patients targets the underlying pathophysiology of the disease to restore orexin signaling.
  • Based on these results, and in consultation with global health authorities, Takeda plans to initiate global Phase 3 trials of TAK-861 in NT1 rapidly in the first half of its fiscal year 2024.
  • Results from the Phase 2b trials have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023).

Global Narcolepsy Drugs Market Research Report 2024: Pipeline Analysis, Key Company Profiles and Recent Developments 2023-2030 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, January 30, 2024

The "Global Narcolepsy Drugs Market (By Therapeutic Type, Disease Type, End User, Regional Analysis), Pipeline Analysis, Key Company Profiles and Recent Developments - Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Narcolepsy Drugs Market (By Therapeutic Type, Disease Type, End User, Regional Analysis), Pipeline Analysis, Key Company Profiles and Recent Developments - Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.
  • The global narcolepsy drugs market has witnessed significant growth in recent years owing to the rising prevalence of narcolepsy and increased awareness about the condition.
  • Latin America and Middle East & Africa are competing closely to grab the maximum share of the global narcolepsy drugs market.
  • Global Narcolepsy Drugs Market - Key Company Profiles, Recent Developments, Financial Insights

Los Angeles Center for Ear, Nose, Throat and Allergy Welcomes Sleep Medicine Specialist, Dr. Cyrus Haghighian, M.D. to Practice

Retrieved on: 
Thursday, February 1, 2024

LOS ANGELES, Feb. 1, 2024 /PRNewswire/ -- The Los Angeles Center for Ear, Nose, Throat, and Allergy (LA CENTA) has announced the addition of Dr. Cyrus Haghighian to their practice. Dr. Haghighian is board certified with a robust educational background and extensive training in sleep conditions.

Key Points: 
  • LOS ANGELES, Feb. 1, 2024 /PRNewswire/ -- The Los Angeles Center for Ear, Nose, Throat, and Allergy (LA CENTA) has announced the addition of Dr. Cyrus Haghighian to their practice.
  • With a specialty in Sleep Medicine, Dr. Haghighian strives to help patients achieve better sleep and overall well-being.
  • Plus, his specialty in sleep medicine is a sought-after expertise that we are lucky to be able to offer."
  • To further advance his expertise he went on to pursue a fellowship in Sleep Medicine at the University of Southern California.

Kleine-Levin Syndrome Foundation Relaunches Medical Advisory Board

Retrieved on: 
Tuesday, January 30, 2024

SAN JOSE, Calif., Jan. 30, 2024 /PRNewswire/ -- The Kleine-Levin Syndrome Foundation today relaunched its Medical Advisory Board to help accelerate scientific understanding about KLS. The volunteer members of the Medical Advisory Board encourage scientific research to advance the search for a cause and a cure for KLS, provide objective medical knowledge to build awareness about KLS, and offer guidance for KLS patients and their caregivers.

Key Points: 
  • Members help encourage scientific inquiry; provide patients evidence-based information and guidance
    SAN JOSE, Calif., Jan. 30, 2024 /PRNewswire/ -- The Kleine-Levin Syndrome Foundation today relaunched its Medical Advisory Board to help accelerate scientific understanding about KLS.
  • "The KLS Foundation Board of Directors is grateful for the extensive knowledge and expertise of these respected researchers and clinicians," said Kleine-Levin Syndrome Foundation President Steve Maier, who experienced 18 KLS episodes from age 13-27.
  • "The Medical Advisory Board will provide the evidence-based guidance sought by so many KLS patients and their caregivers."
  • The relaunched Medical Advisory Board is composed of experts who volunteer their time and knowledge in many medical disciplines relating to KLS, including sleep disorders, neurology, and psychiatry.

Orphan designation: N-{(2S,3R)-4,4-Difluoro-1-(2-hydroxy-2-methylpropanoyl)-2-[(2,3',5'-trifluoro[1,1'-biphenyl]-3-yl)methyl]pyrrolidin-3-yl}ethanesulfonamide Treatment of narcolepsy, 08/11/2023 Positive

Retrieved on: 
Sunday, February 4, 2024

Key facts

Key Points: 
  • Key facts
    - Active substance
    - N-{(2S,3R)-4,4-Difluoro-1-(2-hydroxy-2-methylpropanoyl)-2-[(2,3',5'-trifluoro[1,1'-biphenyl]-3-yl)methyl]pyrrolidin-3-yl}ethanesulfonamide
    - Intended use
    - Treatment of narcolepsy
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2851
    - Date of designation
    - Sponsor
    Takeda Pharma A/S
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Orphan designation: sodium oxybate Treatment of narcolepsy, 13/10/2023 Positive

Retrieved on: 
Sunday, February 4, 2024

EU/3/23/2846 - orphan designation for treatment of narcolepsy

Key Points: 
  • EU/3/23/2846 - orphan designation for treatment of narcolepsy
    sodium oxybate
    OrphanHuman
    Avadel Ireland
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Avadel Pharmaceuticals Announces Strong LUMRYZ Launch Performance and Provides Preliminary Fourth Quarter and Full Year 2023 Financial Highlights

Retrieved on: 
Monday, January 8, 2024

DUBLIN, Ireland, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, today provided a business update including preliminary estimates of fourth quarter and full year net revenue and cash, cash equivalents and marketable securities.

Key Points: 
  • “2023 was transformational for Avadel defined by significant growth and continued execution of milestones critical to Avadel’s success, beginning with the FDA approval and receipt of Orphan Drug Exclusivity for LUMRYZ.
  • The LUMRYZ launch has thus far been marked by robust demand and overwhelmingly positive feedback from the narcolepsy community, health care providers and payers.
  • Launch Progress Through December 31, 2023:
    Greater than 1,900 patients enrolled in Avadel’s RYZUP patient support services:
    More than 1,000 patients initiated therapy.
  • Approximately $19 million and $28 million of net product revenue, respectively, estimated for the quarter and year ended December 31, 2023.

Axsome Therapeutics Provides Preliminary Fourth Quarter and Full Year 2023 Net Revenue and 2024 Anticipated Milestones

Retrieved on: 
Thursday, January 4, 2024

“Axsome completed its first full year as a commercial company in 2023 and, during that time, our medicines reached approximately 100,000 unique patients.

Key Points: 
  • “Axsome completed its first full year as a commercial company in 2023 and, during that time, our medicines reached approximately 100,000 unique patients.
  • Auvelity® net product sales are expected to be approximately $49 million and $130 million for the fourth quarter and full year of 2023, respectively.
  • Sunosi® net product revenue is expected to be $22 million and $74 million for the fourth quarter and full year of 2023, respectively.
  • Net product revenue excludes $66 million in license revenue recognized by Axsome in the first quarter of 2023.

The study of dreams: Scientists uncover new communication channels with dreamers

Retrieved on: 
Wednesday, January 3, 2024

In his sci-fi film Inception (2010), Christophe Nolan imagined his protagonist slipping into other people’s dreams and even shaping their contents. But what if this story wasn’t so far away from real life? Our research suggests that it is possible to interact with volunteers while they are asleep, and even to converse with them at certain key moments.The scientific study of dreamsResearch shows we remember on average one to three dreams per week.

Key Points: 


In his sci-fi film Inception (2010), Christophe Nolan imagined his protagonist slipping into other people’s dreams and even shaping their contents. But what if this story wasn’t so far away from real life? Our research suggests that it is possible to interact with volunteers while they are asleep, and even to converse with them at certain key moments.

The scientific study of dreams

  • Research shows we remember on average one to three dreams per week.
  • However, not everyone is equal when it comes to recalling dreams.
  • Whether people remember their dreams depends on many factors such as gender (women remember their dreams more frequently than men), one’s interest in dreams, as well as the way dreams are collected (some might find it handy to keep track of them with a “dream journal” or a recorder, for example).
  • The private and fleeting nature of dreams makes it tricky for scientists to capture them.
  • Moreover, to understand what happens in the brain while dreaming – and what purpose this activity serves – we would need to be able to compare brain activity during times when dreams occur with those when they are absent.
  • It is therefore imperative to determine precisely when dreams occur in order to further the science of dreams.

Lucid dreaming

  • Lucid dreamers, on the other hand, have the unique ability to remain aware of the dreaming process during REM sleep, a stage of sleep during which brain activity is closer to that of the waking phase.
  • The existence of lucid dreaming has been known since ancient times, but for a long time it was considered esoteric and unworthy of scientific exploration.
  • These two researchers set out to prove that lucid dreamers were indeed asleep when they realised they were dreaming.
  • It is now possible to combine such experiments with brain imaging techniques to study the brain regions involved in lucid dreaming.

From fiction to reality: talking to the dreamer

  • In other words, is it possible to talk to a lucid dreamer?
  • To find out, we exposed a lucid dreamer to tactile stimuli while he was asleep.
  • But the fact that they sometimes did (18% of cases in our study) opened a communication channel between experimenters and dreamers.
  • However, lucid dreaming remains a rare phenomenon and even lucid dreamers are not lucid all the time or throughout REM sleep.

Expanding the communication portal

  • This disease, which causes involuntary sleep, sleep paralysis and an early onset of the REM phase, is associated with an increased propensity for lucid dreaming.
  • We asked them to smile or frown to signal whether the word had been made up or not.
  • Unsurprisingly, people with narcolepsy were able to respond when they were lucid in REM sleep, confirming our results from 2021.
  • By refining the brain markers that predict the moments of connection with the outside world, it should be possible to further optimise communication protocols in the future.


Les auteurs ne travaillent pas, ne conseillent pas, ne possèdent pas de parts, ne reçoivent pas de fonds d'une organisation qui pourrait tirer profit de cet article, et n'ont déclaré aucune autre affiliation que leur organisme de recherche.